The company designed the study based on feedback from the US Food and Drug Administration (FDA). It compared CS014 with valproic acid (VPA), a well-established HDAC inhibitor with a long history of clinical use in neurological conditions such as epilepsy. By showing how the two compounds relate, Cereno aims to build on the extensive existing data on VPA instead of repeating parts of a longer development program.

The open-label, randomized crossover trial was conducted in Sweden with 14 healthy volunteers. Each participant received both CS014 and VPA for seven days in separate treatment periods. The relationship between the two drugs’ plasma concentration profiles at steady state was consistent across all 14 sampling points and matched the assumptions behind the bridging strategy, meeting the company’s predefined expectations. All participants completed the study, all adverse events were mild, and no serious adverse events occurred. More comprehensive results are to be published in a peer-reviewed journal in 2027.

“These positive results address an important pharmacokinetic question and will form part of the total data package for our planned IND submission and discussions with the FDA regarding progression to Phase IIb,” says Rahul Agrawal, CMO and Head of R&D at Cereno Scientific.

Cereno plans to submit an Investigational New Drug (IND) application to the FDA in the fourth quarter of 2026. The placebo-controlled Phase IIb trial is expected to start in the third quarter of 2027, subject to regulatory approval. PH-ILD is a serious, progressive condition with few treatment options – only one therapy is approved in the US and none in Europe.

“Subject to FDA approval, this pathway could allow us to avoid a Phase IIa trial and further non-clinical safety studies, potentially reducing development time, cost, and complexity,” says Sten R. Sörensen, CEO at Cereno Scientific.

CS014 complements Cereno’s lead candidate CS1, which is currently being evaluated in the global Phase IIb EPIMODE trial in pulmonary arterial hypertension (PAH).